On 22 October 2007, orphan designation (EU/3/07/484) was granted by the European Commission to Centre Hospitalier Universitaire de Nantes, France, for adenovirus associated viral vector serotype 4 containing the human RPE65 gene for treatment of Leber's congenital amaurosis.
The sponsorship was transferred to Horama SAS, France, in May 2015.
In November 2016 HoramaSAS changed name to Horama SA.
The sponsor’s name was changed from Horama SAS to Coave Therapeutics and the address was updated in August 2022.
Adenovirus associated viral vector serotype 4 containing the human RPE65 gene
Treatment of Leber's congenital amaurosis
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Documents related to this orphan designation evaluation
EU/3/07/484: Public summary of positive opinion for orphan designation of adenovirus associated viral vector serotype 4 containing the human RPE65 gene for treatment of Leber's congenital amaurosis (PDF/123.48 KB)
First published: 22/01/2008
Last updated: 27/05/2015
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: